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Proteostasis, Astellas Join up on Genetic Disease Drugs

Proteostasis Therapeutics got a new CEO a few months ago; now the Cambridge, MA-based startup has a new development deal to work on, and a new high-profile investor too.

Japanese drugmaker Astellas Pharma inked a partnership today with Proteostasis to try to discover and develop new drugs for an unspecified genetic disease and, potentially, other unnamed disease types. Proteostasis is getting an upfront check of an undisclosed amount . The deal includes a lot of “bio-bucks,” however: downstream payments that could materialize should Proteostasis hit a variety of development milestones.

Specifically, Proteostasis could get up to $400 million in payments if it hits a series of targets on a drug the two companies will develop together. Astellas also has the option to start up two additional programs with Proteostasis on the same terms. So in theory, if all three development projects hit all of their goals, Proteostasis stands to get as much as $1.2 billion. It also has an option to split the rights, both domestically and internationally, to the drug candidates in the partnership.

Astellas has also made an unspecified equity investment in Proteostasis as part of the deal.

The two companies will try to harness Proteostasis’s work in the field of protein homeostasis, or the ability of cells to properly manufacture or deactivate proteins. When this process is upset, like through genetic disorders or aging, proteins do things they aren’t supposed to do, which can lead to diseases. Proteostasis is one of a handful of companies, including Forma Therapeutics, trying to use drugs that can reestablish homeostasis after it’s been perturbed.

Proteostasis is still far away from proving itself. The company’s lead drug candidate, for cystic fibrosis, is in preclinical development. But the company has made a few strategic moves over the past year. First, in December, it added Biogen Idec (NASDAQ: BIIB) as an investor as part of a deal to develop drugs for neurodegenerative diseases. Then, in June, it named Meenu Chhabra, the former head of Seattle-based Allozyne (which is now defunct), its new president and CEO. Proteostasis’s previous CEO was Mark Enyedy, who left the company to become the head of Shire’s internal medicine unit in August 2013—he’s now Shire’s head of corporate development.

With Astellas on board, Proteostasis now has three pharmaceutical companies in its investor syndicate—the others being Sanofi (via Sanofi-Genzyme BioVentures) and Novartis. Elan, which is now owned by Perrigo, is also an investor.

As for the Astellas deal, the two companies aim to make drugs that modulate what’s known as the “unfolded protein response,” a cellular stress reaction that occurs when proteins in the endoplasmic reticulum don’t fold properly. Proteostasis says this happens in a number of genetic, neurodegenerative, and other diseases. The first program will zero in on an unspecified genetic disease.

Proteostasis started up in 2008 with a big $45 million round from HealthCare Ventures, Fidelity Biosciences, New Enterprise Associates, Novartis Option Fund, and Genzyme.

Ben Fidler is Xconomy's Deputy Biotechnology Editor. You can e-mail him at bfidler@xconomy.com Follow @benthefidler